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About the Regulatory Profession

The regulatory function is vital in making safe and effective healthcare products available worldwide. Individuals who ensure regulatory compliance and prepare submissions, as well as those whose main job function is clinical affairs or quality assurance are all considered regulatory professionals.

Regulatory Code of Ethics

One of our most valuable contributions to the profession is the Regulatory Code of Ethics. The Code of Ethics provides regulatory professionals with core values that hold them to the highest standards of professional conduct.

Regulatory Competency Framework

Like all professions, regulatory is based on a shared set of competencies. The Regulatory Competency Framework describes the essential elements of what is required of regulatory professionals at four major career and professional levels.

Regulatory Convergence

Join the brightest minds in regulatory at the annual Regulatory Convergence. See the global regulatory community in action. Intensive workshops. Topical sessions. Meet ups with regulators. This is where it all comes together.

EMA: Orphan Drug Submissions Expected to Increase in 2013

The European Medicines Agency (EMA) announced Thursday that it expects orphan drug submissions to increase slightly in 2013, continuing a multi-year trend that has seen an increase of nearly 40%.

Background: Orphan Drugs in the EU

The 14 February 2013 announcement, made as part of a series of posts foreshadowing the activities of the agency's main advisory committees in 2013, explains the increasing role of EMA's Committee for Orphan Medicinal Products (COMP), the committee charged with reviewing orphan drug products.

Orphan drug products are intended to treat life-threatening or debilitating diseases that affect less than one in 2,000 patients every year, or a disease for which a product would be unable to recoup its investment.

Alternately, products intended to treat disease areas for which there is no "satisfactory method of diagnosis, prevention or treatment" may also be designated as an orphan product under the theory those afflicted with the condition are "orphans" of the drug development process. The latter category of product must, however, be able to show "significant benefit" to the patient.

The product classes are a relatively new innovation in the world of regulatory, emerging in the US in 1983 under the Orphan Drug Act and in the EU in 1999 under the Orphan Regulation (No. 141/2000).

A Growing Trend

That same act established the COMP in the EU. In EMA's announcement, the agency noted that COMP's role is growing-and fast. In 2011, for example, the committee designated 107 submissions as orphan products. In 2012, that number skyrocketed up to 148-38% above the year prior. While 2013 isn't likely to exhibit the same trend, EMA said it expects to receive "more than 150" during the year. It also noted it is already off to a brisk start, with 19 applications received thus far. In 2012 at this time, it had only received 14 applications, it said.

But more than just approvals, EMA also explained that it's working with more groups and patient organizations as well, including EUnetHTA, a health technology assessment (HTA) group focused on reimbursement, and the European Organisation for Rare Diseases (EURORDIS), a patient advocacy group for those suffering from rare or neglected diseases.

With the HTA bodies in particular, EMA said the interactions between regulators and payors will be useful in "sharing information for the common benefit of patients affected by rare diseases and the financial sustainability of the healthcare system."

"The work of the COMP is evolving and constantly adapting to better serve and address patients' needs, the growing scientific knowledge on rare diseases, and the regulatory framework," added Bruno Sepodes, COMP chair.